Laronidase

The global mucopolysaccharidosis treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth is driven by advancements in biotechnology, enhanced diagnostic methods, increased awareness, and the availability of enzyme replacement therapy. Clinical trials for innovative treatments are expanding, despite high costs and lack of reimbursement policies limiting market growth.

The global mucopolysaccharidosis (MPS) treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth drivers include advancements in biotechnology, enhanced diagnostic methods, increased awareness of rare diseases, and the availability of enzyme replacement therapy (ERT). Clinical trials and new drug approvals, such as Lysogene's LYS-SAF302 and JCR Pharmaceuticals' JR-141, are pivotal. Despite high treatment costs and reimbursement challenges, the market is set to expand, with ERT remaining the preferred treatment.

Sanofi reports 15.7% Q3 sales growth, driven by vaccine sales and strong business performance, raising 2024 EPS guidance. Dupixent sales up 23.8%, with full-year target confirmed at €13 billion. Pharma launches grew 67.1%, led by ALTUVIIIO, Nexviazyme, and Rezurock. Vaccines sales increased 25.5%, boosted by flu sales and Beyfortus. Opella sales rose 7.9%, supported by Qunol acquisition. R&D expenses grew 12.7%, while SG&A expenses grew 6.4%. Business EPS at €2.86, up 12.2% reported and 17.6% at CER. Four regulatory approvals and four positive phase 3 data readouts achieved. Negotiations underway to transfer a controlling stake of Opella to CD&R. 2024 business EPS guidance upgraded to low single-digit growth at CER.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is ranked 11th among the best biotech stocks to invest in according to hedge funds. The company focuses on treatments for rare diseases, with a robust product line and a strong internal pipeline. Despite challenges in patient diagnosis and commercialization costs, analysts are optimistic about its long-term profitability. The global biotech industry is expected to grow significantly, driven by innovation and demographic trends, with AI potentially revolutionizing drug discovery. BioMarin's Q2 2024 revenue rose 20% to $712 million, and the company raised its 2024 revenue forecast to between $2.75 billion and $2.825 billion.